Factors Affecting Tacrolimus Concentrations in Children with β-Thalassemia Major receiving HSCT

Article

ASTCT recently published an article in their journal Nucleus detailing the impact of genotype and voriconazole co-administration on tacrolimus trough concentration in pediatric β-thalassemia major patients following allogeneic hematopoietic stem cell transplantation.

The following was recently published in ASTCT’s Nucleus publication. Follow @ATSTCT for the latest on all-things cellular therapy.

In a study published in Pharmacogenomics and Personalized Medicine, researchers investigated the impacts of genotype and voriconazole co-administration on tacrolimus (TAC) trough concentration (C0) in pediatric β-thalassemia major (β-TM) patients following allogeneic hematopoietic stem cell transplantation (allo-HSCT). The authors' findings stress the importance of tailoring drug administration based on patients' genotype profiles, as well as tracking patients' TAC concentrations when administering voriconazole.

For patients receiving allo-HSCT, there is a risk of developing graft versus host disease (GVHD) following transplantation. β-TM patients are therefore at risk of GVHD, as allo-HSCT is currently the sole procedure for curing β-TM. TAC has been utilized as a prophylactic following allo-HSCT, but the effects of its concentration alongside patients' genotypes and co-administration of voriconazole has not been well studied.

Patients were administered TAC two days before allo-HSCT, with venous blood samples collected to determine concentrations before and after transplantation. The results concluded that variations in enzymes such as CYP3A4 greatly impacted TAC concentrations when administered orally. Moreover, the co-administration of voriconazole was found to increase TAC concentrations, necessitating lower TAC doses. Lastly, TAC C0 was found to not be highly correlated with efficacy in these patients, suggesting that C0 is not suitable as an indicator of the efficacy of TAC.

Reference

Li C, Lu J, Zhou S, et al. Influential factors and efficacy analysis of tacrolimus concentration after allogeneic hematopoietic stem cell transplantation in children with β-thalassemia major. Pharmgenomics Pers Med. 2021;14:1221-1237. Published 2021 Sep 24. doi:10.2147/PGPM.S325103

Recent Videos
According to John Henson, MD, “What we need are better treatments to control the [brain] tumor once it’s detected.”
First-degree relatives of patients who passed away from pancreatic cancer should be genetically tested to identify their risk for the disease.
Destigmatizing cancer care for incarcerated patients may help ensure that they feel supported both in their treatment and their humanity.
A lower percentage of patients who were released within 1 year of incarceration received guideline-concurrent care vs incarcerated patients.
A collaboration between the Connecticut Departments of Health and Corrections and the COPPER Center aimed to improve outcomes among incarcerated patients.
Computational models help researchers anticipate how ADCs may behave in later lines of development, while they are still in the early stages.
ADC payloads with high levels of potency can sometimes lead to higher levels of toxicity, which can eliminate the therapeutic window for patients with cancer.
According to Greg Thurber, PhD, target-mediated uptake is the biggest driver of efficacy for antibody-drug conjugates as a cancer treatment.
Co-hosts Kristie L. Kahl and Andrew Svonavec highlight what to expect at the 43rd Annual Chemotherapy Foundation Symposium, such as new chemotherapeutics and targeted therapies.
In neuroendocrine tumor management, patients with insulinoma may be at risk of severe hypoglycemia following receipt of GLP-1 receptor agonists.
Related Content
Rising cancer diagnoses in the US highlight urgent needs for improved oncology education, workforce distribution, and care infrastructure, especially in rural areas.

A Decline in the US Oncology Workforce: What Will the Next Decade Bring?

Julie M. Vose, MD, MBA
December 2nd 2025
Article

Rising cancer diagnoses in the US highlight urgent needs for improved oncology education, workforce distribution, and care infrastructure, especially in rural areas.

Decision-Making Capacity: The Ethical Core of Patient-Centered Oncology

Decision-Making Capacity: The Ethical Core of Patient-Centered Oncology

Daniel C. McFarland, DO;Louis P. Voigt, MD;Yesne Alici, MD
November 24th 2025
Podcast

Daniel C. McFarland, DO, is joined by Louis P. Voigt, MD, and Yesne Alici, MD, who focused on decision-making capacity and patient-centered care.

PAS-004 was deemed safe and tolerable when given in 37 mg capsules, so the trial will proceed to administer the agent in 45 mg capsules.

Cohort 7 of Novel MEK Inhibitor Trial Completed for MAPK Pathway-Driven Tumors

Tim Cortese
November 25th 2025
Article

PAS-004 was deemed safe and tolerable when given in 37 mg capsules, so the trial will proceed to administer the agent in 45 mg capsules.

Experts discussed supportive care and why it should be integrated into standard oncology care.

How Supportive Care Methods Can Improve Oncology Outcomes

Declan Walsh, MD;Denise B. Reynolds, RD;Adam Friedman, MD, FAAD;Daniel C. McFarland, DO;Michelle Fingeret, PhD;Christian J. Nelson, PhD;William S. Breitbart, MD
November 10th 2025
Podcast

Experts discussed supportive care and why it should be integrated into standard oncology care.

Innate lymphoid cells prevent graft-versus-host disease via IL-9-driven T-cell senescence

Innate lymphoid cells prevent graft-versus-host disease via IL-9-driven T-cell senescence

American Society for Transplantation and Cellular Therapy
November 24th 2025
Article

Researchers have observed that HSC–derived innate lymphoid cells prevent GVHD by inducing interleukin 9 driven T-cell senescence.

Tandem CAR T cells targeting mesothelin and MUC16 overcome tumor heterogeneity by targeting one antigen at a time

Tandem CAR T cells targeting mesothelin and MUC16 overcome tumor heterogeneity by targeting one antigen at a time

American Society for Transplantation and Cellular Therapy
November 24th 2025
Article

Researchers have demonstrated that a tandem CAR T cell targeting mesothelin and MUC16 ectodomain is able to outmaneuver tumor heterogeneity, outperforming single target CAR T cells in mixed ovarian and pancreatic models.